The present invention provides duplexed parvovirus vector genomes that are capable under appropriate conditions of forming a double-stranded molecule by intrastrand base-pairing. Also provided are duplexed parvovirus particles comprising the vector genome. Further disclosed are templates and methods for producing the duplexed vector genomes and duplexed parvovirus particles of the invention. Methods of administering these reagents to a cell or subject are also described. Preferably, the parvovirus capsid is an AAV capsid. It is further preferred that the vector genome comprises AAV terminal repeat sequences.
The present invention provides duplexed parvovirus vector genomes that are capable under appropriate conditions of forming a double-stranded molecule by intrastrand base-pairing. Also provided are duplexed parvovirus particles comprising the vector genome. Further disclosed are templates and methods for producing the duplexed vector genomes and duplexed parvovirus particles of the invention. Methods of administering these reagents to a cell or subject are also described. Preferably, the parvovirus capsid is an AAV capsid. It is further preferred that the vector genome comprises AAV terminal repeat sequences.
Richard Samulski - Chapel Hill NC, US Douglas McCarty - Chapel Hill NC, US
International Classification:
C12Q001/70 C12N007/00
US Classification:
435/005000, 435/235100, 435/320100
Abstract:
The present invention provides duplexed parvovirus vector genomes that are capable under appropriate conditions of forming a double-stranded molecule by intrastrand base-pairing. Also provided are duplexed parvovirus particles comprising the vector genome. Further disclosed are templates and methods for producing the duplexed vector genomes and duplexed parvovirus particles of the invention. Methods of administering these reagents to a cell or subject are also described. Preferably, the parvovirus capsid is an AAV capsid. It is further preferred that the vector genome comprises AAV terminal repeat sequences.
Products And Methods For Delivery Of Polynucleotides By Adeno-Associated Virus For Lysosomal Storage Disorders
Douglas M. McCarty - Pataskala OH, US Haiyan Fu - Pataskala OH, US
Assignee:
NATIONWIDE CHILDREN'S HOSPITAL INC. - Columbus OH
International Classification:
A61K 48/00 A61P 3/00 C12N 15/63
US Classification:
424 932, 4353201
Abstract:
The present invention relates to methods and materials useful for systemically delivering polynucleotides across the blood brain barrier using adeno-associated virus as a vector. For example, the present invention relates to methods and materials useful for systemically delivering α-N-acetylglucosamidinase polynucleotides to the central and peripheral nervous systems, as well as the somatic system. Use of these methods and materials is indicated, for example, for treatment of the lysosomal storage disorder mucopolysaccharidosis IIIB. As another example, the present invention relates to methods and materials useful for systemically delivering N-sulphoglucosamine sulfphohydrolase polynucleotides to the central and peripheral nervous systems, as well as the somatic system. Use of this second type of methods and materials is indicated, for example, for treatment of the lysosomal storage disorder mucopolysaccharidosis IIIA.
The University of North Carolina at Chapel Hill - Chapel Hill NC
International Classification:
C12N 15/864
US Classification:
4353201
Abstract:
The present invention provides duplexed parvovirus vector genomes that are capable under appropriate conditions of forming a double-stranded molecule by intrastrand base-pairing. Also provided are duplexed parvovirus particles comprising the vector genome. Further disclosed are templates and methods for producing the duplexed vector genomes and duplexed parvovirus particles of the invention. Methods of administering these reagents to a cell or subject are also described. Preferably, the parvovirus capsid is an AAV capsid. It is further preferred that the vector genome comprises AAV terminal repeat sequences.
Stanley H Schwartz MD 12980 Frederick St STE I, Moreno Valley, CA 92553 9519243244 (phone), 9512436976 (fax)
Languages:
English Spanish
Description:
Mr. Mccarty works in Moreno Valley, CA and specializes in Internal Medicine. Mr. Mccarty is affiliated with Parkview Community Hospital Medical Center, Riverside Community Hospital and San Gorgonio Memorial Hospital.
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Douglas Mccarty
Tagline:
Single dad in Southern Maryland, loving life and having fun